Molecular Therapy Journal

MDA-supported investigators found that intramuscular injections of microdystrophin genes improved muscle health in dystrophin-deficient dogs, a response not previously seen in large animals or humans

posted on January 15, 2013 - 2:34pm

For the first time, gene therapy using a highly miniaturized dystrophin gene resulted in significant improvement in muscle structure and function in dogs with a disorder mimicking human Duchenne muscular dystrophy.